A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)

October 24, 2023 updated by: Regeneron Pharmaceuticals

Clinical Characteristics and Mortality in Patients With Fibrodysplasia Ossificans Progressiva (FOP): A Non-Interventional US National Retrospective Cohort

Primary Objective(s):

  • To describe the demographic characteristics of people living with FOP
  • To describe the prevalence of clinical characteristics of interest in people living with FOP
  • To describe the use of key medications in people living with FOP
  • To estimate the crude mortality rate among people with FOP

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

131

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • New York
      • Tarrytown, New York, United States, 10591
        • Regeneron

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patients with a confirmed diagnosis of FOP in the United States

Description

Inclusion Criteria:

  1. Confirmed FOP diagnosis
  2. Members from the International Fibrodysplasia Ossificans Progressiva Association (iFOPA) cohort who have opted-in to be tokenized by Health Verity Marketplace
  3. Must be enrolled in the closed medical claims data source for any duration of time between January 1, 2018 and December 31, 2022

Exclusion Criteria:

1. Failure to meet inclusion criteria

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Sub-Cohort 1
All patients with active enrollment (closed claims) during part or all the study period.
Other: Non-Interventional
No study treatment will be administered on this study.
Sub-Cohort 2
All patients with active enrollment (closed claims) augmented with open claims
Other: Non-Interventional
No study treatment will be administered on this study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Demographic characteristics
Time Frame: Up to 5 years
Up to 5 years
Prevalence of cardiovascular disease
Time Frame: Up to 5 years
Up to 5 years
Prevalence of metabolic disease
Time Frame: Up to 5 years
Up to 5 years
Prevalence of hematology disease
Time Frame: Up to 5 years
Up to 5 years
Prevalence of infection
Time Frame: Up to 5 years
Up to 5 years
Prevalence of respiratory disease
Time Frame: Up to 5 years
Up to 5 years
Prevalence of reproductive disease
Time Frame: Up to 5 years
Up to 5 years
Prevalence of neurologic disorders
Time Frame: Up to 5 years
Up to 5 years
Prevalence of skeletal disorders
Time Frame: Up to 5 years
Up to 5 years
Prevalence of gastrointestinal disease
Time Frame: Up to 5 years
Up to 5 years
Prevalence of deafness
Time Frame: Up to 5 years
Up to 5 years
Prevalence of dermatologic disorders
Time Frame: Up to 5 years
Up to 5 years
Prevalence of neoplasms
Time Frame: Up to 5 years
Up to 5 years
Average number of prescriptions per FOP patient per year
Time Frame: Up to 5 years
Up to 5 years
Cumulative number of prescriptions per FOP patient
Time Frame: Up to 5 years
Up to 5 years
Incidence of all-cause death
Time Frame: Up to 5 years
Up to 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Regeneron Pharmaceuticals

Investigators

  • Study Director: Clinical Trial Management, Regeneron Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 29, 2023

Primary Completion (Actual)

September 22, 2023

Study Completion (Actual)

September 29, 2023

Study Registration Dates

First Submitted

September 11, 2023

First Submitted That Met QC Criteria

September 26, 2023

First Posted (Actual)

October 3, 2023

Study Record Updates

Last Update Posted (Actual)

October 25, 2023

Last Update Submitted That Met QC Criteria

October 24, 2023

Last Verified

October 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • R2477-FOP-2143

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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